Rare Disease Business Not Such An Easy Road - Just Ask Pfizer

By John Mylant: The rare disease drug business can provide a windfall for pharmaceutical companies in their research as they develop drugs for diseases that are not very common in the general population. But it is not easy getting there. Pfizer (PFE) is a good example of that, as it found out with its drug Tafamidis.There's a reason why many drug companies have an interest in the rare disease business. There are many advantages, such as:

Profits can be higher, since clinical trials for rare disease drugs are usually shorter and have fewer patients (this lowers costs).

The Orphan Drug Act in the U.S. provides a 50% tax credit for the development of drugs that treat diseases affecting fewer than 200,000 patients.

Drugs for rare diseases can find a quicker road to U.S. Food and Drug Administration approval.

Over the past five years, 22% of orphan drugs submitted to the FDA gainedComplete Story »

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By Brian L. Wilson:This year, one of many trends in the world of biotech investing is the increasing popularity of orphan drugs on Wall Street.An orphan drug is a special designation that is given to drugs that are intended to treat very rare diseases (in the United States this means less than 200,000). In the past we've seen drug companies avoid the targeting of certain diseases because of the very small number of patients that would be providing sales revenue, but things have changed.

For many years, pharmaceutical research strategy was all about chasing big blockbusters in huge markets like diabetes and heart disease. Now, many of those drugs have lost patent protection, and companies have lost billions in sales to generic competitors.

ByPropThink:Thursday morning, DARA Biosciences (DARA) announced it has submitted an application to the U.S. FDA requesting Orphan Drug status for KRN5500 for the treatment of chronic chemotherapy-induced peripheral neuropathy (CCIPN).

You may know about priority review vouchers (PRV).
Designed by Duke University economists as a means of encouraging drug development for unaddressed tropical diseases, PRVs were made law in 2007.
Essentially, they are transferrable go-to-the-front-of-the-regulatory-line “chits.” And they are awarded to companies that develop approved drugs for certain orphan diseases. Estimates of the financial value of these chits range from $200 million to well over half a billion dollars. They’re like golden tickets.

NEW DELHI (AP) — India's Supreme Court on Monday rejected drug maker Novartis AG's attempt to patent an updated version of a cancer drug in a landmark decision that health activists say ensures poor patients around the world will get continued access to cheap versions of lifesaving medicines.